Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

The recruitment status of this study is unknown because the information has not been verified recently.
Verified October 2003 by Office of Rare Diseases (ORD).
Recruitment status was  Active, not recruiting
Information provided by:
Office of Rare Diseases (ORD) Identifier:
First received: June 2, 2000
Last updated: June 23, 2005
Last verified: October 2003

OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory cytokines and for the production of nitric oxide by alveolar macrophages to identify possible risk factors for pulmonary complications.

II. Investigate the underlying mechanism for the development of significant pulmonary complications in these patients during HSCT.

III. Evaluate bronchoalveolar lavage fluid and serum obtained from these same patients at the time a pulmonary complication develops post-HSCT, or at 60 days post-HSCT if there has been no pulmonary complications.

I Cell Disease
Globoid Cell Leukodystrophy
Niemann-Pick Disease
Pulmonary Complications
Mucopolysaccharidosis I
Mucopolysaccharidosis VI
Metachromatic Leukodystrophy
Gaucher's Disease
Wolman Disease

Study Type: Observational
Study Design: Primary Purpose: Screening

Resource links provided by NLM:

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U.S. FDA Resources

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 10
Study Start Date: August 1999
Detailed Description:


Patients undergo bronchoscopy with bronchoalveolar lavage (BAL) prior to allogeneic hematopoietic stem cell transplantation (HSCT). ELISA assays for cytokines are performed. Patients are followed post-HSCT for the development of transplant related pulmonary complications. A repeat bronchoscopy with BAL is performed at the time pulmonary complications develop or at day 60 post-HSCT if no complications develop. Cytokine assays are repeated.


Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
  • Diagnosis of an inborn error of metabolism eligible for allogeneic hematopoietic stem cell transplantation on protocol UMN-MT-1995-01
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00005900

United States, Minnesota
Fairview University Medical Center
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Fairview University Medical Center
Study Chair: K. Scott Baker Fairview University Medical Center
  More Information

No publications provided Identifier: NCT00005900     History of Changes
Other Study ID Numbers: 199/15111, UMN-MT-1999-18, UMN-MT-9818
Study First Received: June 2, 2000
Last Updated: June 23, 2005
Health Authority: Unspecified

Keywords provided by Office of Rare Diseases (ORD):
Gaucher's disease
I cell disease
Niemann-Pick disease
Wolman disease
disease-related problem/condition
genetic diseases and dysmorphic syndromes
globoid cell leukodystrophy
inborn errors of metabolism
metachromatic leukodystrophy
mucopolysaccharidosis I
mucopolysaccharidosis VI
oncologic disorders
pulmonary complications
rare disease

Additional relevant MeSH terms:
Gaucher Disease
Leukodystrophy, Globoid Cell
Leukodystrophy, Metachromatic
Mucopolysaccharidosis I
Mucopolysaccharidosis VI
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Niemann-Pick Diseases
Wolman Disease
Adrenal Gland Diseases
Adrenal Insufficiency
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Carbohydrate Metabolism, Inborn Errors
Central Nervous System Diseases
Cholesterol Ester Storage Disease
Connective Tissue Diseases
Demyelinating Diseases
Endocrine System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hereditary Central Nervous System Demyelinating Diseases
Heredodegenerative Disorders, Nervous System
Histiocytosis, Non-Langerhans-Cell
Infant, Newborn, Diseases
Intellectual Disability processed this record on March 26, 2015